量化儿科肺动脉高压治疗的副作用和照护者负担。
Quantifying side effects and caregiver burdens of pediatric pulmonary hypertension therapies.
机构信息
Department of Public Health, Brigham Young University, 2148 LSB, Provo, UT, 84660, USA.
Emma Eccles Jones College of Education & Human Services, Utah State University, Logan, UT, USA.
出版信息
BMC Pediatr. 2023 Jan 25;23(1):43. doi: 10.1186/s12887-023-03860-2.
BACKGROUND AND OBJECTIVES
Pulmonary hypertension (PH) is a rare, but serious disease among children. However, PH has been primarily evaluated among adults. Consequently, treatment therapies have not been fully evaluated among pediatric populations and are used in an 'off label' manner. The purpose of this study was to estimate the side effect profiles of the most commonly prescribed pediatric PH therapies and to understand the burdens placed upon families caring for children living with PH.
METHODS
Participants were recruited online through the "Families of children with pulmonary hypertension" Facebook group and asked to complete a survey about PH treatments.
RESULTS
A total of 139 parents of a child living with PH completed the survey. Almost all children used ≥ 1 medication to treat PH, with 52% using ≥ 3 medications. The highest average number of side effects was reported by users of Treprostinil, Selexipag and type-5 phosphodiesterase (PDE) inhibitors. The most common side effects were skin flushing, headache, nasal congestion, joint/muscle pain, and nausea. In terms of accessing care, 81% travel ≥ 20 miles and 68% travel for ≥ 60 min to receive care.
CONCLUSIONS
We found an array of treatment combinations employed to mitigate symptoms of PH in children, with a wide range of side effects. We also found a large, unseen economic, emotional, and time burden of caring for a child living with PH. Further research is warranted to understand the clinical implications of these side effects to move towards labeled usage of these therapies rather than post-hoc off-label usage.
背景与目的
肺动脉高压（PH）在儿童中较为罕见，但较为严重。然而，PH 主要在成人中进行评估。因此，儿科人群并未充分评估治疗疗法，并且以“超说明书”的方式使用。本研究旨在评估最常用于治疗儿童 PH 的药物的副作用，并了解照顾患有 PH 的儿童的家庭所面临的负担。
方法
通过“肺动脉高压儿童家庭”的 Facebook 群组在线招募参与者，并要求他们完成有关 PH 治疗的调查。
结果
共有 139 名患有 PH 的儿童的父母完成了调查。几乎所有的儿童都使用了≥1 种药物来治疗 PH，其中 52%的儿童使用了≥3 种药物。使用前列环素、塞来昔帕和 5 型磷酸二酯酶（PDE）抑制剂的患者报告的平均副作用最高。最常见的副作用是皮肤潮红、头痛、鼻塞、关节/肌肉疼痛和恶心。在获取治疗方面，81%的人旅行≥20 英里，68%的人旅行≥60 分钟以接受治疗。
结论
我们发现有多种治疗组合用于减轻儿童 PH 的症状，但副作用范围广泛。我们还发现，照顾患有 PH 的儿童会带来巨大的、未被看到的经济、情感和时间负担。需要进一步研究以了解这些副作用的临床意义，从而转向这些疗法的标签使用，而不是事后超说明书使用。
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